Safety and Efficacy of CLBS03 in Adolescents With Recent Onset Type 1 Diabetes (The Sanford Project T-Rex Study)

Lisata Therapeutics

Status and phase: Completed; Phase 2

Conditions: Diabetes Mellitus

Treatments: Biological: CLBS03 High Dose; Biological: CLBS03 Low Dose; Biological: Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT02691247

CLBS03-P01

Details and patient eligibility

About

This clinical trial will explore the safety and effect of autologous ex vivo expanded polyclonal regulatory T-cells on beta cell function in patients, aged 8 to 17, with recent onset T1DM. Other measures of diabetes severity and the autoimmune response underlying T1DM will also be explored. Eligible subjects will receive a single infusion of CLBS03 (high or low dose) or placebo.

Enrollment: 113 patients

Sex: All

Ages: 8 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and females aged 8 to 17 years of age
• Diagnosis of T1DM within 100 days of receipt of study drug
• Positive for at least one islet cell autoantibody
• Peak MMTT-stimulated C-peptide level > 0.2 pmol/mL (at the screening visit)
• Weight of ≥30 kg
• Must agree to use a reliable and acceptable method of contraception for the duration of participation
• Willing and medically acceptable to postpone live vaccine immunizations for one year after infusion
• Written informed consent and written assent

Exclusion criteria

• Hemoglobin less than the lower limit of normal
• Leukocytes <3,000/μL; neutrophils <1,500/μL; lymphocytes <800/μL; platelets <100,000/μL
• Regulatory T-cells present in peripheral blood at <20 cells per μL
• Current or ongoing use of non-insulin pharmaceuticals (that may affect glycemic control)
• Current or anticipated use of systemic corticosteroids or other immunomodulatory drugs
• Recent serious bacterial, viral, fungal, or other opportunistic infections
• History of malignancy or serious uncontrolled cardiovascular, nervous system, pulmonary, renal, or gastrointestinal disease
• Serologic evidence of current or past viral infection: human immunodeficiency virus (HIV), Hepatitis B, Hepatitis C, and human T-lymphotropic virus (HTLV) 1/2
• Positive QuantiFERON® tuberculosis (TB) test, purified protein derivative (PPD) skin test, history of tuberculosis, or active TB infection
• Active infection with Epstein-Barr Virus or Cytomegalovirus
• Liver disease
• Pregnant or breast-feeding
• Vaccination with a live virus within 8 weeks of receipt of study drug
• Vaccination with a killed virus within 2 weeks of receipt of study drug
• Participation in an investigational drug study within 90 days prior to screening
• Previously treated with a T-Reg based cell therapy
• History of allergy to gentamicin

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Factorial Assignment

Masking: Quadruple Blind

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113 participants in 3 patient groups, including a placebo group

CLBS03 Low Dose

Experimental group

Description:

A single infusion of CLBS03 Low Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP (United States Pharmacopoeia) infusion solution.

Treatment:

Biological: CLBS03 Low Dose

CLBS03 High Dose

Experimental group

Description:

A single infusion of CLBS03 High Dose, a cell product comprised of autologous, ex vivo expanded regulatory T-cells resuspended in sterile USP infusion solution.

Treatment:

Biological: CLBS03 High Dose

Placebo

Placebo Comparator group

Description:

A single infusion of placebo, consisting of the infusion solution only

Treatment:

Biological: Placebo